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Treatment of severe primary IGF-1 deficiency using rhIGF-1 preparation — first three years of Polish experience
Affiliations- Department of Paediatrics, Endocrinology, Diabetology, Metabolic Disorders, and Cardiology of Developmental Age, Pomeranian Medical University, Szczecin, Poland
- Department of Endocrinology and Diabetology of Children, and Adolescents, Wroclaw Medical University, Wroclaw, Poland
- Department of Paediatrics, Diabetology, and Endocrinology, Medical University of Gdansk, Gdansk, Poland
- Department of Paediatrics, Endocrinology, and Diabetes, Medical University of Silesia, Katowice, Poland
- Division of Clinical Genetics, Department of Molecular Biology, and Genetics, School of Medicine in Katowice, Medical University of Silesia, Katowice, Poland
- Department of Basic Medical Sciences, Wroclaw Medical University, Wroclaw, Poland
- Department of Paediatric Endocrinology, and Diabetology, Medical University Children Hospital, Lublin, Poland
- Department of Endocrinology and Metabolic Diseases, Polish Mother’s Memorial Hospital — Research Institute, Lodz, Poland
- Department of Paediatric Endocrinology, Medical University of Lodz, Lodz, Poland
- Department of Endocrinology and Metabolic Diseases, Medical University of Lodz, Lodz, Poland, Poland
- Department of Paediatrics, Medical University of Silesia, Katowice, Poland
- Department of Paediatrics, Endocrinology, Diabetology with Cardiology Division, Medical University of Bialystok, Bialystok, Poland
- Department of Paediatrics and Endocrinology, Medical University of Warsaw, Warsaw, Poland
- Department of Clinical Auxology and Paediatric Nursing, Poznan University of Medical Sciences, Poznan, Poland
- Department of Endocrinology and Diabetology, The Children’s Memorial Health Institute, Warsaw, Poland
- The Faculty of Medicine and Health Sciences, Jan Kochanowski University, Kielce, Poland
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Abstract
Introduction: The objective of this study was to analyse the effects of the first three years of treatment with recombinant human insulinlike growth factor 1 (rhIGF-1) in patients from the Polish population.
Material and methods: Twenty-seven children (22 boys and five girls) aged 2.8 to 16.0 years old were qualified for treatment with rhIGF-1 (mecasermin) in different treatment centres, according to Polish criteria: body height below –3.0 SD and IGF-1 concentration below percentile 2.5 with normal growth hormone (GH) levels. Mecasermin initial dose was 40 μg/kg bw twice a day and was subsequently increased to an average of 100 μg/kg bw twice a day. Body height, height velocity, weight, body mass index (BMI), and adverse events were measured.
Results: Mecasermin treatment resulted in a statistically significant increase in body height (1.45 ± 1.06 SD; p < 0.01) and height velocity in comparison with pre-treatment values. The biggest change in height velocity happened during the first year and diminished during subsequent years. Body weight and BMI also increased significantly after treatment (1.16 ± 0.76 SD and 0.86 ± 0.75 SD, respectively; p < 0.01). Eight patients reported adverse events. These were mild and temporary and did not require treatment modification except in two patients.
Conclusions: Treatment with rhIGF-1 was effective and safe in Polish patients with primary IGF-1 deficiency. It had a clear beneficial effect on the height of the patients and significantly accelerated the height velocity, particularly in the first year of treatment.
Abstract
Introduction: The objective of this study was to analyse the effects of the first three years of treatment with recombinant human insulinlike growth factor 1 (rhIGF-1) in patients from the Polish population.
Material and methods: Twenty-seven children (22 boys and five girls) aged 2.8 to 16.0 years old were qualified for treatment with rhIGF-1 (mecasermin) in different treatment centres, according to Polish criteria: body height below –3.0 SD and IGF-1 concentration below percentile 2.5 with normal growth hormone (GH) levels. Mecasermin initial dose was 40 μg/kg bw twice a day and was subsequently increased to an average of 100 μg/kg bw twice a day. Body height, height velocity, weight, body mass index (BMI), and adverse events were measured.
Results: Mecasermin treatment resulted in a statistically significant increase in body height (1.45 ± 1.06 SD; p < 0.01) and height velocity in comparison with pre-treatment values. The biggest change in height velocity happened during the first year and diminished during subsequent years. Body weight and BMI also increased significantly after treatment (1.16 ± 0.76 SD and 0.86 ± 0.75 SD, respectively; p < 0.01). Eight patients reported adverse events. These were mild and temporary and did not require treatment modification except in two patients.
Conclusions: Treatment with rhIGF-1 was effective and safe in Polish patients with primary IGF-1 deficiency. It had a clear beneficial effect on the height of the patients and significantly accelerated the height velocity, particularly in the first year of treatment.
Keywords
insulin-like growth factor-1 (IGF-1); growth hormone insensitivity; IGF-1 deficiency; short stature; growth-promoting therapy; mecasermin; Poland
About this articleTitle
Treatment of severe primary IGF-1 deficiency using rhIGF-1 preparation — first three years of Polish experience
Journal
Issue
Article type
Original paper
Pages
20-27
Published online
2018-10-23
Page views
3143
Article views/downloads
1787
DOI
Pubmed
Bibliographic record
Endokrynol Pol 2019;70(1):20-27.
Keywords
insulin-like growth factor-1 (IGF-1)
growth hormone insensitivity
IGF-1 deficiency
short stature
growth-promoting therapy
mecasermin
Poland
Authors
Elżbieta Petriczko
Tomasz Jackowski
Anita Horodnicka-Józwa
Beata Wikiera
Anna Noczyńska
Maria Korpal-Szczyrska
Dorota Birkholz-Walerzak
Ewa Małecka-Tendera
Barbara Kalina-Fraska
Maria Kalina
Ewa Barg
Iwona Beń-Skowronek
Leszek Szewczyk
Maciej Hilczer
Joanna Smyczyńska
Renata Stawerska
Andrzej Lewiński
Katarzyna Ziora
Artur Bossowski
Edyta Pietrewicz
Beata Pyrżak
Andrzej Kędzia
Mieczysław Szalecki
Agnieszka Kilian
Mieczysław Walczak
References (13)- Rosenbloom AL. Mecasermin (recombinant human insulin-like growth factor I). Adv Ther. 2009; 26(1): 40–54.
- Filus A, Zdrojewicz Z. Insulinopodobny czynnik wzrostu-1 (IGF-1) — budowa i rola w organizmie człowieka [Insulin-like growth factor-1 (IGF-1) — structure and the role in the human body. Pediatr Endocrinol Diabetes Metab. 2015; 20(4): 161–169.
- Iranpoor H, Omidinia E, Vatankhah V. Expression of Recombinant Human Insulin-like Growth Factor Type 1 (rhIGF-1) in Escherichia coli. Avicenna J Med Biotechnol. 2015; 7(3): 101–105.
- Petriczko E, Wikiera B, Horodnicka-Józwa A, et al. A two year observation of the process of applying recombinant IGF-1 to treat short stature in children with primary IGF-1 deficiency — case reports of 3 patients. Pediatr Endocrinol Diabetes Metab. 2011; 17(4): 233–238.
- Backeljauw P, Bang P, Clayton PE. Diagnosis and management of primary insulin-like growth factor-I deficiency: current perspectives and clinical update. Pediatr Endocrinol Rev. 2010; 7 Suppl 1: 154–171.
- Hardin DS. Statement 6: patients who are unresponsive to GH therapy should be given a trial of IGF-I. Pediatr Endocrinol Rev. 2008; 5(Suppl 3): 857–860.
- Coutant R, Dörr HG, Gleeson H. Diagnosis of endocrine disease: limitations of the IGF1 generation test in children with short stature. Eur J Endocrinol. 2012; 166(3): 351–357.
- Ranke MB, Savage MO, Chatelain PG. Long-term treatment of growth hormone insensitivity syndrome with IGF-I. Results of the European Multicentre Study. The Working Group on Growth Hormone Insensitivity Syndromes. Horm Res. 1999; 51(3): 128–134.
- Chernausek SD, Backeljauw PF, Frane J. Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrinol Metab. 2007; 92(3): 902–910.
- Guevara-Aguirre J, Rosenbloom AL, Vasconez O. Two-year treatment of growth hormone (GH) receptor deficiency with recombinant insulin-like growth factor I in 22 children: comparison of two dosage levels and to GH-treated GH deficiency. J Clin Endocrinol Metab. 1997; 82(2): 629–633.
- Richmond EJ, Rogol AD. Recombinant human insulin-like growth factor-I therapy for children with growth disorders. Adv Ther. 2008; 25(12): 1276–1287.
- Midyett LK, Rogol AD, Van Meter QL, et al. MS301 Study Group. Recombinant insulin-like growth factor (IGF)-I treatment in short children with low IGF-I levels: first-year results from a randomized clinical trial. J Clin Endocrinol Metab. 2010; 95(2): 611–619.
- Ahn SY, Pi LQ, Hwang ST, et al. Effect of IGF-I on Hair Growth Is Related to the Anti-Apoptotic Effect of IGF-I and Up-Regulation of PDGF-A and PDGF-B. Ann Dermatol. 2012; 24(1): 26–31.
